Meet Gaven, an Actual AGAMREE^® (vamorolone) Patient

This is a sponsored post written by Catalyst Pharmaceuticals, Inc.

by Catalyst Pharmaceuticals, Inc. | August 19, 2025

Insights Into Living With Duchenne Muscular Dystrophy (DMD)

Gaven lives in Texas, sharing a home with his mother, father, and 12-year-old brother, Tristan. Like many 10-year-old boys, he enjoys visits to the museum, going to restaurants, and most of all, riding motorcycles. His parents describe him as logical and quick, with a dry sense of humor. He is resilient and an adventure seeker. Like only 1 in every 5,000 males born in the United States, Gaven is living with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle weakness.

Gaven received his diagnosis when he was 5½ years old. For his parents, Jessica and Manuel, the news rocked their world. As they slowly started to rebuild, a new hope grew, one focused on being positive and making every little thing count. They understood, maybe better than most, that every day is something to be cherished.

At about this same time, a once-in-a-lifetime opportunity presented itself, one that sparked a fire in Gaven. A long-distance motorcycle rider named Riot reached out. Speaking to Jessica, he expressed his interest in dedicating a day to Gaven, a way to increase awareness of DMD. That contact quickly grew into something far more special. Riot and Gaven forged a deep friendship. This bond afforded Gaven the chance to ride, something that gave him a sense of freedom and the opportunity to remember that there is more to life than DMD.

After his diagnosis, Gaven started treatment with a generic corticosteroid. When side effects caused him and his family concern, they tried another treatment. Soon, he experienced different issues—including difficulty focusing and behavioral symptoms. With limited options available at the time, they switched back to prednisolone.

“He would come home in tears because our extremely smart child was now staring at the wall during math class because he couldn’t focus. He started having tantrums, similar to what a toddler would have over the smallest thing you can imagine.”

– Jessica, mother of Gaven

In 2024, Gaven and his parents learned about AGAMREE^®. AGAMREE is approved to treat DMD in patients as young as 2 years old. Following a discussion with his healthcare provider, they decided to switch. After Gaven started AGAMREE, his mother asked him what he thought. Gaven said, “I’m not switching back. I feel better.” He told his mother that his tummy didn’t hurt as much and he felt better, overall.

“I can’t tell you exactly why I feel better, but my legs don’t hurt as bad.”

– Gaven, an actual AGAMREE patient

Patients may switch to AGAMREE from another oral corticosteroid without treatment interruption or period of prior corticosteroid dosage reduction. It is important to always take AGAMREE as prescribed and to tell your healthcare provider about all medical conditions, and about all medications the patient takes.

“Be willing to try new things. For us, trying new things was AGAMREE, and it’s worked in our favor.”

– Jessica, mother of Gaven

Out of Gaven’s relationship with Riot, something special emerged. Together, they helped found Mile Monsters, Inc., a group of long-distance motorcycle endurance riders who raise money and help build awareness for DMD. By trying something new, Gaven harnessed his passion and found his voice.

If you or a member of your family is living with DMD, consider talking to your healthcare provider about AGAMREE. To learn more about AGAMREE, visit AGAMREE.com.

To hear more about Gaven and his family, as well as stories from other real AGAMREE patients, click here.

What is AGAMREE?

AGAMREE is a prescription medicine used to treat Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

IMPORTANT SAFETY INFORMATION

Patients should not take AGAMREE if they are allergic to vamorolone or any of the inactive ingredients in AGAMREE.

What is the most important information I should know about AGAMREE?

Do not stop AGAMREE, or change the amount taken, without first checking with your healthcare provider. There may be a need for gradual dose reductions to decrease the risk of adrenal insufficiency crisis, which can be fatal.
There is an increased risk of infection when taking corticosteroids like AGAMREE. Tell your healthcare provider if the patient has had recent or ongoing infections or has recently received a vaccine. Seek immediate medical advice in the case of fever or other signs of infection. Some infections can be severe, and sometimes fatal. Patients should avoid exposure to chickenpox or measles; alert your healthcare provider immediately if exposure occurs.
Corticosteroids, including AGAMREE, can cause an increase in blood pressure and water retention. Your healthcare provider may monitor for these increases during treatment.
There is an increased risk of developing a hole in the stomach or intestines in patients with certain gastrointestinal disorders when taking corticosteroids like AGAMREE.
Corticosteroids, including AGAMREE, can cause severe behavioral and mood changes. Seek medical attention if behavioral or mood changes develop.
There is a risk of osteoporosis with prolonged use of corticosteroids like AGAMREE, which can lead to vertebral and long bone fractures.
Corticosteroids like AGAMREE may cause cataracts or glaucoma. Your healthcare provider should monitor for these conditions if AGAMREE treatment continues for more than 6 weeks.
Immunizations should be up to date according to immunization guidelines prior to starting therapy with AGAMREE. Live‑attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting AGAMREE. Live‑attenuated or live vaccines should not be administered in patients taking AGAMREE.
Rare instances of severe allergic reaction have occurred in patients receiving corticosteroid therapy.

Before taking AGAMREE, tell your healthcare provider about all medical conditions, including if the patient:

has decreased liver function.
is pregnant or planning to become pregnant. AGAMREE can harm an unborn baby.
is breastfeeding or planning to breastfeed. AGAMREE may appear in breastmilk and could affect a nursing child.

Certain medications can cause an interaction with AGAMREE. Tell your healthcare provider about all the medicines the patient takes, including prescription and over-the-counter medicines, dietary supplements, and herbal products.

What are the possible side effects of AGAMREE?

The most common side effects with AGAMREE include facial puffiness (cushingoid features), psychiatric disorders, vomiting, weight gain, and vitamin D deficiency. These are not all the possible side effects of AGAMREE.

Call your doctor for medical advice about side effects.

Please see full Prescribing Information for additional Important Safety Information.

To report SUSPECTED ADVERSE REACTIONS, contact Catalyst Pharmaceuticals, Inc. at 1-844-347-3277 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.